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CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...

CRISPR and crRNAs

CRISPR and crRNAs

Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...

Homologous Recombination

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...

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Related Experiment Video

Updated: Jun 1, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

Current progress in CRISPR-Cas systems for rare diseases.

Juveriya Israr¹, Ajay Kumar²

¹Institute of Biosciences and Technology, Shri Ramswaroop Memorial University, Barabanki, Uttar Pradesh, India.

Progress in Molecular Biology and Translational Science

|January 17, 2025

Summary

This summary is machine-generated.

CRISPR-Cas gene editing precisely alters genetic code, offering revolutionary treatments for rare diseases. Advanced CRISPR technologies enhance safety and efficacy for genetic therapies.

Keywords:

CRISPER-Cas systems Clinical trials Genome editing Precision medicine Rare diseases Therapeutic applications

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Last Updated: Jun 1, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

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Published on: August 2, 2018

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

Published on: September 2, 2021

Area of Science:

Molecular Biology
Genetics
Biotechnology

Background:

CRISPR-Cas is a bacterial defense system against viruses.
It functions as a programmable DNA-cutting system using guide RNA and Cas enzymes.
This technology enables precise genetic modifications.

Purpose of the Study:

To explore the profound influence of CRISPR-Cas technology on molecular biology and genetics.
To highlight its potential in gene function research, disease modeling, and genetic therapy.
To discuss its transformative impact on biotechnology, agriculture, and personalized medicine.

Main Methods:

Utilizes guide RNA to direct Cas enzymes to specific genomic targets.
Cas enzymes cleave DNA for gene insertion, deletion, or modification.
Advanced methods like base and prime editing allow precise DNA alterations without double-strand breaks.

Main Results:

CRISPR-Cas enables accurate correction of genetic flaws underlying rare diseases.
Offers potential for targeted and effective treatments for rare medical conditions.
Improved safety and precision in gene editing reduce adverse effects.

Conclusions:

CRISPR-Cas technology is revolutionizing genetics and medicine, offering hope for rare disease cures.
Gene therapy using CRISPR-Cas holds promise for treating genetic disorders.
Ongoing research in CRISPR-Cas gene editing, delivery systems, and clinical trials is advancing rare disease treatment.